ORLANDO, FL—Overall survival was significantly longer with ponatinib than allogeneic stem cell transplant in patients with chronic phase chronic myeloid leukemia (CP-CML) who harbor the T315I mutation. The first analysis to confirm these findings was presented at the 57th American Society of Hematology (ASH) Annual Meeting.1

Ponatinib may therefore “be a promising alternative for patients with CP-CML with the T315I mutation in this setting,” said Franck E. Nicolini, MD, PhD, of Centre Hospitalier Lyon Sud in France.

Overall survival was found to be similar between intervention groups in accelerated phase CML (AP-CML) and longer for allogeneic stem cell transplant patients in blast phase CML (BP-CML) and de novo Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL).

Ponatinib is approved in the United States and Europe for the treatment of refractory CML or de novo Ph+ ALL and those with the BCR-ABL1 T315I mutation “or for whom no other TKI therapy is indicated,” Dr. Nicolini said. “Ponatinib represents an alternative treatment option to allogeneic SCT in eligible patients harboring the T315I mutation, but differences in survival between such patients treated with ponatinib and allogeneic SCT have not been studied to date,” he added, in explaining the rationale for the study.

In this study, data from 184 patients harboring the T315I mutation from the phase 2 PACE trial of ponatinib (128 patients) and the European Bone Marrow Transplant registry (56 patients) were pooled and an indirect comparison of ponatinib and allogeneic SCT conducted. Of these patients, 90 were in CP-CML, 26 were in AP-CML, 41 were in blast phase (BP-CML), and 27 had Ph+ ALL.

Time from T315I detection to intervention was significant between the ponatinib and allogeneic SCT groups for CP-CML, AP-CML, and BP-CML (all P < .001), but not for the de novo Ph+ ALL patients (P = .052).

A retrospective analysis of patients with CML determined prevalence of arterial thrombotic events in those receiving nilotinib as first-line treatment and prevalence by patient age. Data were presented at ASH ...